Exploring transparent reporting and data availability in systematic reviews to identify subgroup evidence: imaging for suspected hepatocellular carcinoma in the non-cirrhotic liver.

We aim to illustrate the role of complete and transparent reporting coupled with access to data sourced from published systematic reviews, especially assisting in the identification of evidence for subgroups within the context of a rare disease. To accomplish this principle, we provide a real-world example encountered during the revision of the Dutch clinical practice guideline for hepatocellular carcinoma. Specifically, we retrieved insights from two Cochrane reviews to identify direct evidence concerning the diagnostic test accuracy of computed tomography and magnetic resonance imaging for detecting hepatocellular carcinomas in suspected patients without liver cirrhosis.

Introducing re-weighted range voting in clinical practice guideline prioritization: Development and testing of the re-weighted priority-setting (REPS) tool.

We aimed to develop and test a tool based on the re-weighted range voting mechanism to prioritize items (i.e. key questions) in a priority-setting assessment for clinical practice guidelines.

[How often do medical guidelines refer to articles written in Dutch?].

Objective: For Dutch medical guidelines, Dutch research articles published in the NTvG (NederlandsTijdschriftvoorGeneeskunde) and other medical journals are not searched systematically and are only used sporadically. Using these publications in the process of guideline development can be useful for recommendations regarding the Dutch context of care. In this research, we have investigated how often and in which parts of Dutch guidelines articles published in NTvG are used.

Rare disease registries: potential applications towards impact on development of new drug treatments.

Background: Low prevalence, lack of knowledge about the disease course, and phenotype heterogeneity hamper the development of drugs for rare diseases. Rare disease registries (RDRs) can be helpful by playing a role in understanding the course of the disease, and providing information necessary for clinical trial design, if designed and maintained properly. We describe the potential applications of a RDR and what type of information should be incorporated to support the design of clinical trials in the process of drug development, based on a broad inventory of registry experience.

A systematic review to investigate the measurement properties of goal attainment scaling, towards use in drug trials.

Background: One of the main challenges for drug evaluation in rare diseases is the often heterogeneous course of these diseases. Traditional outcome measures may not be applicable for all patients, when they are in different stages of their disease. For instance, in Duchenne Muscular Dystrophy, the Six Minute Walk Test is often used to evaluate potential new treatments, whereas this outcome is irrelevant for patients who are already in a wheelchair.